Groundbreaking CRISPR treatment for blindness only works for subset of patients
By Jocelyn Kaiser,
Science
| 11. 17. 2022
After some early but cautious optimism, a company is shelving its pioneering gene-editing treatment for a rare inherited blindness disorder. Editas Medicine announced today the trial trying to use the gene editor CRISPR to treat Leber congenital amaurosis 10 (LCA10) led to “clinically meaningful” vision improvements in only three of 14 patients.
In the study, patients received a subretinal injection of a modified virus carrying genetic material encoding components of CRISPR, a DNA-cleaving enzyme, and two RNA strands to guide the protein to its target sequences. For this trial, CRISPR was designed to snip out a problematic part of a gene called CEP290, which encodes a protein needed by the photoreceptor cells that eyes use to sense light. It was the first trial in the world to directly inject the gene-editing system into the body, rather than applying CRISPR to people’s cells in a lab dish and then reinfusing them.
Two of the patients whose vision improved—a woman treated early in the trial and a 14-year-old boy treated more recently, both of whom received midrange doses—had two defective copies...
Related Articles
By Mary Annette Pember, ICT News [cites CGS' Katie Hasson] | 04.18.2025
The sight of a room full of human cadavers can be off-putting for some, but not for Haley Omeasoo.
In fact, Omeasoo’s comfort level and lack of squeamishness convinced her to pursue studies in forensics and how DNA can be...
Gray wolf by Jessica Eirich via Unsplash
“I’m not a scarcity guy, I’m an abundance guy”
– Colossal co-founder and CEO Ben Lamm, The New Yorker, 4/14/25
Even the most casual consumers of news will have seen the run of recent headlines featuring the company Colossal Biosciences. On March 4, they announced with great fanfare the world’s first-ever woolly mice, as a first step toward creating a woolly mammoth. Then they topped that on April 7 by unveiling one...
By Katrina Northrop, The Washington Post | 04.06.2025
photo via Wikimedia Commons licensed under CC by 3.0
China's most infamous scientist is attempting a comeback. He Jiankui, who went to jail for three years after claiming he had created the world's first genetically altered babies, says he remains...
By Anumita Kaur [cites CGS’ Katie Hasson], The Washington Post | 03.25.2025
Genetic information company 23andMe has said that it is headed to bankruptcy court, raising questions for what happens to the DNA shared by millions of people with the company via saliva test kits.
Sunday’s announcement clears the way for a new...